A new report by research group EvaluatePharma has highlighted the strength of the orphan drugs market.
Big pharma
The report indicates that the market for orphan drugs, which are used in the treatment of rare diseases, is expected to grow by 11% over the next five years to $209 billion. Compared to all other prescription medicine sales, the growth of the orphan drugs market is more than double.
The analysis demonstrates the lucrative market of orphan drugs and how large pharmaceutical companies are taking notice of it.
US biotech firm Celgene is set to take the top spot in orphan drug sales by 2022, with other players such as pharma company AstraZeneca, drug maker Abbvie, and healthcare organisation Johnson&Johnson expected to become major names in the market.
The analysis also shows that the median cost of orphan drugs per patient is 5.5 times higher than other drugs. The report indicates that in 2016 the average cost for a US patient, per year for an orphan drug was $140,443, compared with $27,756 for a non-orphan drug.
However the high prices for orphan drugs are a growing concern. This year the UK’s National Institute for Health and Care Excellence (NICE), rejected Alexion’s drug Kanuma for the treatment of lysosomal acid lipase deficiency (LAL-D), a genetic disorder which can lead to multi-organ failure and potential premature death.
NICE argued that the cost of the drug, priced at almost £500,000 per patient, could not be justified by its long-term treatment benefits.
About the decision Professor Carole Longson, NICE health technology evaluation centre director, said: "Throughout this extremely complicated evaluation we have given the company many opportunities to improve the terms under which sebelipase alfa could be offered to patients. However, even their best offer to date falls far short given the considerably uncertainties about its longer term benefits and its very high cost."
In the US the 1983 Orphan Drug Act was passed to incentivise drug makers to create drugs for rare diseases which affect less than 200,000 people. However, a recent paper argues that drug makers are finding ways to exploit the law to widen the market for orphan drugs. The paper argues that pharmaceutical companies are gaining approval for supposed orphan drugs, but the products are used in ways not intended by the FDA and still receive the lucrative orphan drug protections and exclusive benefits.